Title:
GENE EDITING SYSTEM FOR TREATING USHER SYNDROME
Document Type and Number:
WIPO Patent Application WO/2023/191570
Kind Code:
A1
Abstract:
The present invention relates to a gene editing system for treating Usher syndrome, to a disease treatment method using same, and the like, wherein by using the gene editing system of the present invention, an exon 13 region mutated in the Usherin (USH2A) gene is deleted with high efficiency, thereby treating type 2 Usher syndrome effectively. In addition, the gene editing system of the present invention comprises a miniaturized Cas12f1 protein-based endonuclease and an engineered guide RNA having a shorter length and improved indel efficiency, and a carrier having a limited packaging size, such as adeno-associated virus (AAV), can also be used, and therefore, in vivo or intracellular delivery efficiency can also be maximized.
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Inventors:
KIM YONG SAM (KR)
KIM DO YON (KR)
KIM DO YON (KR)
Application Number:
PCT/KR2023/004330
Publication Date:
October 05, 2023
Filing Date:
March 30, 2023
Export Citation:
Assignee:
GENKORE INC (KR)
International Classes:
C12N9/12; C12N9/22; C12N15/10; C12N15/113; C12N15/86
Foreign References:
| US20210115419A1 | 2021-04-22 | |||
| KR20210053228A | 2021-05-11 | |||
| US20210403905A1 | 2021-12-30 | |||
| KR20190058358A | 2019-05-29 |
Other References:
PENDSE NACHIKET, LAMAS VERONICA, MAEDER MORGAN, PAWLYK BASIL, GLOSKOWSKI SEBASTIAN, PIERCE ERIC A., CHEN ZHENG-YI, LIU QIN: "Exon 13-skipped USH2A protein retains functional integrity in mice, suggesting an exo-skipping therapeutic approach to treat USH2A-associated disease", BIORXIV, 4 February 2020 (2020-02-04), pages 1 - 34, XP093095559, DOI: 10.1101/2020.02.04.934240
Attorney, Agent or Firm:
SEUM IP (KR)
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